Groundbreaking cystic fibrosis treatment reaches more patients

(BPT) - Over the past three decades, meaningful progress has been made in the research and understanding of cystic fibrosis (CF).¹ While thousands of patients were able to benefit from this work, there are still people with CF who are ineligible for existing treatments.^2,3,4,5 This is a driving force behind the serial innovation in this space. Now, more individuals with CF could benefit from ALYFTREK^® (vanzacaftor/tezacaftor/deutivacaftor) (10 mg/50 mg/125 mg tablets; 4 mg/20 mg/50 mg tablets)-the latest Food & Drug Administration (FDA)-approved, once-daily cystic fibrosis transmembrane conductance regulator (CFTR) modulator developed by Vertex Pharmaceuticals-thanks to the treatment recently being approved to support even more patients with CF.^6,7

ALYFTREK is a prescription medicine for people six years and older who have a diagnosis of CF and at least one genetic change (variant) in theCFTRgene that is responsive to ALYFTREK or results in protein production.⁶ Talk to your healthcare provider to learn if you have an indicated CF gene variant. If your genotype is unknown, an FDA-cleared CF variant test should be used to confirm the presence of the indicated variant(s).⁶

Advancing treatment for cystic fibrosis

Cystic fibrosis is a rare, life-shortening, progressive genetic disease caused by variants in the CFTR gene that affect the respiratory system, digestive system and other organs.^8,9 This gene was only discovered in 1989, leading to innovative work in the CF space that focused on targeting the underlying gene dysfunction.¹ This led to the development of a new class of medicine-CFTR modulator therapies.^1,10

Today, CFTR modulator therapies from Vertex Pharmaceuticals, including ALYFTREK, are treating over 75,000 people with CF in over 60 countries on six continents.^11,12 This population represents approximately two-thirds of diagnosed people with CF who are eligible for CFTR modulator therapies.¹¹

"With more than 2,000 variants of the CFTRgene that cause cystic fibrosis, this label expansion brings ALYFTREK, our most advanced and our only once-daily therapy, to even more people with CF variants," said Meg Duncan, Head of U.S. Medical Affairs-CF at Vertex.^6,13 "This is the heart of our ambition-to serially innovate in hopes of bringing our transformative therapies to more people living with CF and give them the opportunity to live life without limits."

A novel approach for rare disease innovation

While there are thousands of CFTR variants, over 80% of CF patients have one in common-known as a F508del variant.¹ There are another 10% to 18% of patients who have a rare CFTR variant.^1,4 In most cases, there are so few patients with each rare variant that a clinical trial is incredibly difficult to organize. A final approximately 7% do not make CFTR protein at all, making this population even more challenging to reach.⁴

In an effort to bring transformative therapies to more people living with CF, Vertex developed assays of Fischer Rat Thyroid (FRT) cells to replicate these rare variants, which allows them to study how the medicines could work for these different patient groups.¹⁴

"For 25 years, we've been dedicated to CF research, including the last decade focused on rare variants, all in partnership with the incredible CF community. Their stories have inspired us to push boundaries, expand the reach of our medicines and make meaningful progress toward helping even more patients," said Duncan. "We're deeply grateful for their role in this journey and remain committed to advancing together."

To learn more about ALYFTREK and treatment eligibility, visit ALYFTREK.com or talk to your doctor.

This article was sponsored by Vertex Pharmaceuticals Incorporated.

What is ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor)?

ALYFTREK is a prescription medicine for people 6 years and older who have a diagnosis of cystic fibrosis (CF) and at least 1 genetic change (variant) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ALYFTREK or results in protein production.

Talk to your healthcare provider to learn if you have an indicated CF gene variant.

It is not known if ALYFTREK is safe and effective in children under 6 years.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about ALYFTREK?

Elevated liver enzymes have been observed in patients taking ALYFTREK. Cases of serious liver damage and liver failure leading to transplantation and death have been seen in some people with or without a history of liver problems taking elexacaftor/tezacaftor/ivacaftor (TRIKAFTA), a medicine which has the same or similar active ingredients as ALYFTREK.

• Your healthcare provider will do blood tests to check your liver:
  • before you start ALYFTREK
  • then every month during your first 6 months
  • then every 3 months during the next 12 months
  • then at least every year while taking ALYFTREK
    
    

• Your healthcare provider may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past or are experiencing signs or symptoms of liver injury.
  
  

• Stop taking ALYFTREK and call your healthcare provider right away if you have any of the following symptoms of liver problems:
  • pain, swelling, or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • mental changes
  • nausea or vomiting
  • dark, amber-colored urine
  • loss of appetite
  • fluid in your stomach area (ascites)

What should I tell my healthcare provider before taking ALYFTREK?

Before taking ALYFTREK, tell your healthcare provider about all of your medical conditions, including if you:

• have or have had liver problems
• are allergic to ALYFTREK or any ingredients in ALYFTREK. See the end of the Medication Guide for a complete list of ingredients in ALYFTREK
• have taken another medicine with elexacaftor, tezacaftor, or ivacaftor before and temporarily or permanently stopped because of side effects. Your healthcare provider may want to see you more often
• have kidney problems
• have or have had mental health problems
• are pregnant or plan to become pregnant. It is not known if ALYFTREK will harm your unborn baby. You and your healthcare provider should decide if you will take ALYFTREK while you are pregnant
• are breastfeeding or planning to breastfeed. It is not known if ALYFTREK passes into your breast milk. You and your healthcare provider should decide if you will take ALYFTREK while you are breastfeeding

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

ALYFTREK may affect the way other medicines work, and other medicines may affect how ALYFTREK works. The dose of ALYFTREK may need to be adjusted when taken with certain medicines. Ask your healthcare provider or pharmacist for a list of these medicines if you are not sure.

Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

What should I avoid while taking ALYFTREK?

• Avoid food or drink that contains grapefruit while you are taking ALYFTREK

What are the possible or reasonably likely side effects of ALYFTREK?

ALYFTREK can cause serious side effects, including:

• See "What is the most important information I should know about ALYFTREK?"
  
  

• Serious allergic reactions can happen to people who are treated with ALYFTREK. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include:
  • rash or hives
  • tightness of the chest or throat or difficulty breathing
  • light-headedness or dizziness
    
    

• Increased pressure around the brain (intracranial hypertension) has happened in people treated with medicines containing the same or similar ingredients as ALYFTREK. If you experience an unusual headache, blurred vision, double vision, or vision loss, call your healthcare provider right away
  
  

• Serious mental health problems, such as anxiety, depression, suicidal thoughts and behaviors, and trouble sleeping, have happened in people treated with ALYFTREK. If you experience new or worsening mental health problems, call your healthcare provider right away
  
  

• Abnormality of the eye lens (cataract) has happened in some children and adolescents treated with ALYFTREK. If you are a child or adolescent, your healthcare provider should perform eye examinations before and during treatment with ALYFTREK to look for cataracts

The most common side effects of ALYFTREK include:

• cough
• pain or swelling of your nose or throat (nasopharyngitis)
• upper respiratory tract infection (common cold), including stuffy and runny nose
• headache
• mouth or throat pain
• flu (influenza)
• tiredness
• increase in liver enzymes
• rash
• sinus congestion

Your healthcare provider should monitor you during treatment with ALYFTREK. You may require additional monitoring if your treatment with a medicine that works like ALYFTREK has been previously stopped or interrupted because of side effects.

Tell your healthcare provider if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects of ALYFTREK. For more information, ask your healthcare provider or pharmacist. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see full Prescribing Information, including Medication Guide with Important Warning.

The news, reports, views and opinions of authors (or source) expressed are their own and do not necessarily represent the views of CRWE World.